Human Autologous Lung Stem Cell Transplant for Idiopathic Pulmonary Fibrosis

Last updated: March 13, 2025
Sponsor: University of North Carolina, Chapel Hill
Overall Status: Suspended

Phase

1

Condition

Idiopathic Pulmonary Fibrosis

Cystic Fibrosis

Lung Injury

Treatment

Lung Spheroid Stem Cells 100 million

Lung Spheroid Stem Cells 200 million

Clinical Study ID

NCT04262167
18-1430
  • Ages 40-80
  • All Genders

Study Summary

Purpose:

To demonstrate the safety and efficacy of autologous Lung Spheroid Stem Cells (LSCs) administered by intravenous infusion in patients with idiopathic pulmonary fibrosis Progressive Fibrotic Interstitial Lung Disease.

Participants:

Patients with Idiopathic Pulmonary Fibrosis (IPF) and Progressive Fibrotic Interstitial Lung Disease

Procedures (methods):

24 patients previously diagnosed with idiopathic pulmonary fibrosis or Progressive Fibrotic Interstitial Lung Disease meeting all inclusion/exclusion criteria will be evaluated at baseline. LSCs will be grown from autologous trans-bronchial pulmonary biopsy specimens. The first group, consisting of 6 patients will be randomized after completion of the screening procedures to either a treatment group of 100 million LSCs administered via intravenous infusion or to a control group (standard care) in a 2:1 LSC to control group ratio. The second group of 18 patients will be randomized after completion of the screening procedures to either a treatment group of 200 million LSCs administered via intravenous infusion or to a control group (standard care) in a 2:1 LSC to control group ratio. Patients will be randomized using permuted blocks in a 2:1 LSC to control group ratio, providing a distribution of 8:4:12 patients among the control, low dose, and high dose groups, respectively. If the patient is randomized and 100 million LSCs are not achieved, then the patient will be analyzed separately and another patient enrolled. Intravenous infusion of LSCs will take place 4-8 weeks after the pulmonary biopsies are obtained. All patients will be followed up at months 0.5, 1, 3, 6, 9, 12, 18, and 24 after infusion to complete the safety and efficacy assessments listed herein. All patients will receive standard of care for their IPF.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Male or female between the ages of 40 to 80.

  • Diagnosis of a Progressive Fibrotic Interstitial Lung Disease

  • Diagnosis of IPF based on the following criteria in accordance with AmericanThoracic Society (ATS) guidelines for diagnosing IPF:

  1. Definite usual interstitial pneumonia (UIP) confirmed on surgical lung biopsy (SLB) with all other etiologies for UIP excluded OR High resolution CT scan (HRCT) showing definite UIP with all other etiologies for UIP excluded.

  2. Probable UIP on both imaging and surgical lung biopsy with all other etiologiesfor UIP excluded.

  • Forced vital capacity (FVC) greater than 50% of predicted with a ratio of forcedexpiratory volume in 1 second to FVC (FEV1/FVC) greater than 0.75 (Pulmonaryfunction tests must be completed no more than 90 days before screening).

  • Diffusing capacity for carbon monoxide (DLCO) greater than 25% of predictedcapacity.

  • Ability to perform a 6-Minute Walk Test (6MWT) at screening.

  • Competency to understand the information given in the Human Research and EthicsCommittee (HREC) approved Informed Consent Form and must sign the form prior to theinitiation of any study procedures

Exclusion

Exclusion Criteria:

  • Diagnosis of an interstitial lung disease (ILD) or restrictive lung disease otherthan IPF or Progressive Fibrotic Interstitial Lung Disease.

  • Obstructive lung disease as determined by evidence of airflow obstruction on HRCT orphysiologic criteria including: FEV1/FVC ratio less than 0.75, Residual volume (RV)greater than 120% by plethysmography or significant (verified by radiologist)emphysema on HRCT or evidence of reactive airway disease by change in FEV1 ofgreater than 12% following bronchodilator challenge.

  • Evidence of sustained improvement lung function defined as improvement frompre-therapy pulmonary function tests (PFTs) observed with two or more successivepost-therapy PFTs over the year prior to randomization.

  • Active or recent (less than 60 days prior to enrollment) significant respiratorytract infections, or a history of frequent (greater than 2 per year for the last 2years) infective exacerbations of IPF.

  • Hospitalization within 60 days of screening for an acute exacerbation of IPF (AE-IPF).

Chronic heart failure (NYHA class III/IV) or known left ventricular ejection fraction less than 45%.

  • Acute or chronic impairment (other than dyspnea) which limits the ability to complywith study requirements and procedures including the 6MWT.

  • Subject requires hemodialysis, peritoneal dialysis or hemofiltration.

  • Infection with HIV

  • Viral Hepatitis

  • Resting oxygen requirements or >4 L of nasal canula oxygen needed with exertion

Study Design

Total Participants: 24
Treatment Group(s): 2
Primary Treatment: Lung Spheroid Stem Cells 100 million
Phase: 1
Study Start date:
October 22, 2020
Estimated Completion Date:
December 31, 2027

Connect with a study center

  • University of North Carolina as Chapel Hill

    Chapel Hill, North Carolina 27514
    United States

    Site Not Available

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